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Stealth BioTherapeutics' Submission of New Drug Application for Elamipretide Accepted by FDA for Barth Syndrome Treatment

Tuesday, April 09, 2024

Stealth BioTherapeutics, a biotech firm specializing in developing treatments for conditions associated with mitochondrial dysfunction, has announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for elamipretide, intended to address Barth syndrome. This decision follows positive outcomes from the SPIBA-001 Phase 3 Natural History Control Study and additional supportive data from the TAZPOWER Part 2 baseline-controlled trial. Elamipretide has received Fast Track Designation in 2017, Orphan Drug Designation in 2018, and Rare Pediatric Disease Designation in 2020.

Chair of the Barth Syndrome Foundation, Kate McCurdy, expressed appreciation for the FDA's consideration of patients, families, and advocates, highlighting the urgent need for Barth syndrome treatment.

The FDA plans to convene an advisory committee meeting to discuss the application. Despite the NDA being assigned a standard review designation, the company has requested the FDA to reconsider.

CEO Reenie McCarthy welcomed the FDA's commitment to a transparent review process and expressed readiness to engage with committee experts to emphasize the seriousness of Barth syndrome and the need for an effective treatment.

If approved, elamipretide would be the first marketing authorization for this mitochondrial targeted therapeutic. It is also being developed for primary mitochondrial myopathy, with pivotal data from the Phase 3 NuPOWER trial expected in late 2024.

Barth syndrome is a rare genetic condition characterized by cardiac abnormalities and various health complications, predominantly affecting males. It significantly reduces life expectancy, with most deaths occurring before age five. Currently, there are no FDA- or EMA-approved therapies for Barth syndrome. Elamipretide has received Orphan Drug, Fast Track, and Rare Pediatric Designations from the FDA, as well as Orphan Drug Designation from the EMA for treating Barth syndrome.



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